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Mashup Score: 8
People in England to benefit from groundbreaking one-off gene therapy for severe sickle cell disease approved by NICE
Source: www.nice.org.ukCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 20Abstract - 2 day(s) ago
Click on the article title to read more.
Source: onlinelibrary.wiley.comCategories: General Medicine News, Hem/OncsTweet-
The #EAHAD25 abstracts have been published in the journal Haemophilia and are available online. The meeting is in Milan next week and going by the quality of these abstracts it promises to be a great meeting. https://t.co/XQKnlbh4rT
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Mashup Score: 19Andexanet alfa: trials just leave us with more questions - 24 day(s) ago
ANNEXA-I, the first ever randomised controlled trial of a reversal agent for direct oral anticoagulants, was published in 2024. The trial, which randomised patients with intracranial haemorrhage to andexanet alfa or usual care was mandated by the Federal Drugs Administration as part of its conditional approval in 2018, an approval that was originally based on the single-arm trial, ANNEXA-4. ANNEXA-I was stopped early for benefit and showed a reduction in the number of patients with significant haematoma expansion.
Source: www.rpthjournal.orgCategories: General Medicine News, Hem/OncsTweet-
RT @richardbuka: Pleased to share this perspective on andexanet alfa, now published in @RPTHjournal #openaccess🔓 https://t.co/pl1myt01oI I…
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Mashup Score: 17Andexanet alfa: trials just leave us with more questions - 24 day(s) ago
ANNEXA-I, the first ever randomised controlled trial of a reversal agent for direct oral anticoagulants, was published in 2024. The trial, which randomised patients with intracranial haemorrhage to andexanet alfa or usual care was mandated by the Federal Drugs Administration as part of its conditional approval in 2018, an approval that was originally based on the single-arm trial, ANNEXA-4. ANNEXA-I was stopped early for benefit and showed a reduction in the number of patients with significant haematoma expansion.
Source: www.rpthjournal.orgCategories: General Medicine News, Hem/OncsTweet-
RT @richardbuka: Pleased to share this perspective on andexanet alfa, now published in @RPTHjournal #openaccess🔓 https://t.co/pl1myt01oI I…
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Mashup Score: 2
Sangamo Therapeutics said on Monday that partner Pfizer has terminated their hemophilia A gene therapy co-development agreement, sending its shares down nearly 64% in extended trade.
Source: www.reuters.comCategories: General Medicine News, Hem/OncsTweet-
In a surprise move, Pfizer has terminated its agreement with Sangamo to develop a #hemophilia A gene therapy. I think this says a lot about hemophilia A gene therapy in general. https://t.co/XJGvM0zEN7 https://t.co/0MwBT6eQlg
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Mashup Score: 3Publicly funded US research will have to be published without delay under new policy - 1 month(s) ago
Research funded by the US National Institutes of Health (NIH) will have to be made publicly available without a period of delay,1 under the agency’s new public access policy. The policy, which is open to consultation until 21 February 2025, has been designed to accelerate access to publicly funded research results as part of the institute’s commitment to transparency and accessibility. The biggest change from the NIH’s existing policy, produced in 2008, is that the 12 month embargo period before manuscripts resulting from NIH funding must be made publicly available will be removed as of 31 December 2025. This meets an expectation set out by the US government in 20222 for publications and supporting data from federally funded research to be made publicly accessible without an embargo. The NIH’s move comes as other research funders across the world make efforts to increase transparency. Earlier this year the European Union removed a deferral mechanism …
Source: www.bmj.comCategories: General Medicine News, Hem/OncsTweet-
The direction of travel is clear. Open access publication from the start is likely to become the dominant player in publishing. Publicly funded research in the US is likely to require immediate open access publication. https://t.co/A7iPt8BzL2 #publishing
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Mashup Score: 49FDA approves drug to prevent or reduce the frequency of bleeding episodes for patients with hemophilia A with inhibitors or hemophilia B with inhibitors - 1 month(s) ago
Action The U.S. Food and Drug Administration has approved Alhemo (concizumab-mtci) for routine prophylaxis to prevent or reduce the frequency of bleeding
Source: www.einnews.comCategories: General Medicine News, Hem/OncsTweet-
The FDA has now also approved Alhemo, the NovoNordisk anti-TFPI for treatment of persons age 12 and over with hemophilia A or B with inhibitors. So now we have two anti-TFPIs approved, one for inhibitor and one for non-inhibitor patients. https://t.co/5PBfe7Xp2e #hemophilia
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Mashup Score: 2
Ruth Jessen Hickman, MD, is a freelance medical and science writer based in Bloomington, Indiana. Population: Roughly 1 in 200,000 people across ethnicities; may present with hemolytic anemia, macrothrombocytopenia, stomatocytes, xanthomas, and/or elevated cholesterol Treatment: Ezetimibe first line; potential adjunctive treatment with bile acid sequestrants and/or statins, other cardiovascular risk management Social media hashtags: #sitosterolemia Resources: sitosterolemiafoundation.org The rare genetic
Source: ashpublications.orgCategories: General Medicine News, Hem/OncsTweet-
Long news item about sitosterolemia in the current issue of ASH clinical news. My experience is that it is more difficult to treat than this article suggests. https://t.co/Gh3sXEuuOl #sitosterolemia
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Mashup Score: 22Adeno-associated virus–based gene therapy for hemophilia A and B: a systematic review and meta-analysis - 2 month(s) ago
Abstract. Adeno-associated virus (AAV)–based gene therapy is an emerging treatment for hemophilia A (HA) and hemophilia B (HB). In this systematic review a
Source: ashpublications.orgCategories: General Medicine News, Hem/OncsTweet-
Systematic review and meta-analysis of all the AAV gene therapy trials in #hemophilia A and B. Remarkably, 395 patients have had gene therapy for hemophilia during the clinical trials already. https://t.co/zmuSlymezk https://t.co/lbUuAc1PP8
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Mashup Score: 5Sitosterolemia with Orbital Xanthogranulomas | NEJM - 2 month(s) ago
A 50-year-old woman presented with a 5-year history of xanthogranulomas under her eyes. Laboratory studies were notable for thrombocytopenia and very high levels of low-density lipoprotein cholesterol.
Source: www.nejm.orgCategories: General Medicine News, Hem/OncsTweet-
Amazing image of a patient with #sitosterolemia in the @NEJM this week. The blood film with huge platelets and stomatocytes is diagnostic. I have never seen xanthogranulomas in any of our patients with this condition. https://t.co/XLbQ17IcBG https://t.co/dky2gw46pE
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The UK health authorities have agreed to fund gene editing gene therapy for sickle cell disease. Essentially, this means that the technology is not only a significant advance (MHRA approval) but it also represents value for money (NICE approval). https://t.co/HxZu5dJo3V